young patients’ engagement in the€¦ · young patients’ engagement in the ... and empowerment...
Post on 26-Jun-2020
4 Views
Preview:
TRANSCRIPT
Young patients’ engagement in the European Joint
Programme on Rare Diseases –EJPRD
Mariangela Lupo
Can children be involved in thisprocess?
The UN Convention on theRights of the Child, Article12 states Children andYoung People have a rightto have their views heard inall matters affecting themand for these to be takenseriously
Fundamental principles underpinning
the rights of the child in Europe
Principles of the
“Best interests”
“Evolving capacities”
of the child
International Convention
of the Rights of the Child (UN)
Challenges in the engagement of children
Some key factors influencechildren comprehension of theinformation and theconsequent involvement inclinical research process
Age
Physical condition/disease condition
Previous existing knowledge
Language
Mental state
❖ Engaging children early on in theresearch process and educating themabout the importance of participating inclinical research, could help in reducingthe patients’ retention and fosteringtreatments compliance.
❖ A patient-centred approach promotesthe collaboration between the differentactors of the research process and shouldbe included in the best practices of thepaediatric research in order to enhancequality and relevance of the researchitself.
Young patients engagement
❖ Due to the paediatric specific developmental characteristics, specifically tailored methods should be applied to the training and empowerment process of paediatric patients.
❖ Educate and empower the paediatric patients in the specific topic of the project for which their help is requested. For example, improve the language, content and format of the assent document.
❖ Dynamic interactivity to collect feedback aiming to improve the project by using the best methods to collect the information. For example, focus groups, questionnaires or surveys, personal interviews, etc.
Specifically tailored methods should be applied to the training and empowerment process of paediatric patients
European Joint Programme on Rare Diseases
EJP-RD aims to create a research and innovationpipeline “from bench to bedside” ensuring rapidtranslation of research results into clinical applicationsand uptake in healthcare for the benefit of patients.
European Joint Programme on Rare Diseases
• Union contribution: 55 M€ (70% reimbursement rate)• Total budget (min. submitted): 101 M€ (→ expected > 110 M€)• Number of partners: 88• Number of participating countries (beneficiaries and LTPs): 35 including 27 EU MS
(AT, BE, BG, CZ, DE, ES, EE, FI, FR, GR, HU, HR, IE, IT, NL, LT, LV, LU, MT, PL, PT, RO, SE, SK, SL, SV, UK), 7 associated (AM, CH, GE, IL, NO, RS TK) and third countries (CA)
• Timeline: Jan 2019 – Dec 2023
• Types of partners:
o 33 research funding bodies/ministries
o 12 research institutes
o 22 universities/hospital universities
o 11 hospitals
o 5 EU infrastructures (BBMRI, EATRIS, ECRIN, ELIXIR, INFRAFRONTIER) + EORTC
o EURORDIS & ePAGs
o 6 charities/foundations (FTELE, AFM, FFRD, FGB, BSF, IT)
European Joint Programme on Rare Diseases
The specific objective of EJP-RD is to improve integration,efficacy, production and social impact of research on rarediseases through the development, demonstration andpromotion of sharing of research and clinical data, materials,processes, knowledge and know-how, and an efficient model offinancial support for research on rare diseases
The whole programme is based on the
mixture/integration of already existing
activities/programs/tools/trainings and the creation of
new ones (new added value)
1
2 4
3FUNDING ACCELERATING
TRANSLATION OF RESEARCH &
THERAPY
DEVELOPMENT
COORDINATED
ACCESS TO
DATA &
SERVICES
CAPACITY
BUIDLING &
EMPOWERMENT
COORDINATION& TRANSVERSAL ACTIVITIES
INTEGRATIVE RESEARCH STRATEGY
SUSTAINABILITY
ETHICAL & REGULATORY
COMMUNICATION
Coordinated byEJP RD STRUCTURE
WP6
Joint Transnational Calls
WP7
Networking scheme WP 11
Virtual Platfform for data &
resources
WP 10
User-driven strategic planning
for P2
WP 12
Enabling sustainable FAIRness
WP 13
Holistic approaches for rare
disease diagnostics and
therapeutics
WP 19
Facilitating
partnerships and
accelerating translation
WP 20
Validation , use and
development of
innovative
methodologies for
clinical studies
WP 14
Training on data management &
quality
WP 15
Capacity building and training of
patients and researchers
WP 16
Online Academic education
course
WP 17
ERN RD training and support
programme
WP5
COMMUNICATION & DISSEMINATION
WP3
SUSTAINABILITY
WP2
STRATEGY
WP1 COORDINATION & MANAGEMENT
WP8
RDR Challenges
WP9
Monitoring of funded
projects
WP 18
Development and adaptation of
training activities
WP4
ETHICS, LEGAL, REGULATORY & IPR
P1 P2 P3 P4
Pillar 1: funding collaborative research on RD
The Pillar will foster joint transnational calls for collaborative researchprojects resulting in financial support to third parties encompassing variousaspects of rare diseases.
In addition, the financial support actions will be extended to the NetworkingScheme (to encourage sharing of knowledge on rare diseases betweenclinicians, (biomedical) scientists and patient organizations) and the RareDisease Research Challenges (innovative funding scheme to foster private-public partnerships and multistakeholder collaborations).
Pillar 2: Innovative coordinated access to data and services for transformative RD research
The Pillar will develop a FAIR virtual platform for RD informationencompassing research data, samples, tools and standards to support andaccelerate rare diseases research. In addition, pilot and proof-of-conceptprojects will be put in place to enable multidisciplinary, holisticapproaches for rare disease diagnostics and therapeutics and fosteringcreation of complete diseases pathways.
Pillar 3: capacity building and empowerment
The pillar will be raising the level of knowledge and know-how within the RD researchand care community, including through ERNs and RD patient representatives andadvocates. The pillar foresees the development of:
❖ a comprehensive programme of training courses will tackle areas such asresearch data management, orphan drug development, HTA & regulatoryprocesses, best practice guidelines and ERN cross-cutting themes & needs.
❖ training courses will be developed and adapted to address the specific needs ofEU13 countries
❖ first-in-Europe online academic course to provide an EU-wide streamlinededucation programme on RD research to all interested stakeholders.
Pillar 4: accelerating the translation of high potential projects and improving outcomes of clinical studies in
small populationsBy creating a bridge between basic research and medical innovation, otherwise known as the “bench to bedside” approach, this pillar aims to support the RD community to more effectively translate high quality research into high impact interventions for the RD patient community. To this aim:
❖ Research projects funded through Pillar 1 and previously by E-Rare will be selected for their translational potential and specifically supported and guided throughout this process.
❖ Pillar 4 will foster the development of innovative methodologies tailored for clinical studies in RDs by specifically supporting ERNs to use the most adapted methodologies improving trial studies in RDs.
❖ Demonstration studies will be funded to validate existing and most recently presented innovative methodologiesas well as most promising new statistical methodologies;
1
2 4
3FUNDING ACCELERATING
TRANSLATION OF RESEARCH &
THERAPY
DEVELOPMENT
COORDINATED
ACCESS TO
DATA &
SERVICES
CAPACITY
BUIDLING &
EMPOWERMENT
COORDINATION& TRANSVERSAL ACTIVITIES
INTEGRATIVE RESEARCH STRATEGY
SUSTAINABILITY
ETHICAL & REGULATORY
COMMUNICATION
Coordinated byTEDDY INVOLVEMENT
PILLAR 3
WP15: Capacity building & training of patients and researchers in rare diseases
research and processes
WP17: ERN RD training & support programmes
WP16: Online academic education course
WP14: Training on data management & quality
Trainings on: orphanet nomenclature – standards & quality of genetics/genomics
data in clinical practice – strategies to foster undiagnosed diseases – biobanks
sample data management – rare diseases registries & FAIRification at source –
European Rare Diseases Registry Infrastructure
Expert Patients and Researchers EURORDIS Summer school – scientific innovation
and translation research aspects in RDs for patient advocates – leadership &
communication skills for patient advocates and representatives – eduocation
material and activities for paediatric patients
Based on assessed needs of the RD community – in collaboration with universities
– 10 to 12 modules with accreditation – e-learning format open to all – Future
Learn platform
The overall objective of this WP is to address one key component of the EJP on RD that is to improve RD research and innovation and to enhance the uptake of research results by building the capacity of the patient community and other key stakeholders.
Coordinated by
TEDDY LEADS TASK 15.4Educational materials and activities for paediatric patients
The planned activities include short workshops in collaboration with Fundaciò Sant Joan de Deu, EURORDIS and the eYPAGnet (the EuropeanYoung Persons' Advisory Group network) for 15 patients’ representativesfocusing on the following areas:
❖ Genetic /rare disease characteristics and evolution ❖ Role for paediatric patients and relevance of clinical and translational
research (including patients’ engagement in clinical trials, the consent issue, the patient-reported outcomes in the context of paediatric rare diseases)
❖ Orphan Medicinal Products availability and access for paediatricpatients on the EU market, pharmacovigilance and off-label use in paediatric setting
Coordinated by
TEDDY LEADS TASK 15.4Educational materials and activities for paediatric patients
The Face to Face ‘paediatric patient experts training course’ will be hosted in Italy (TEDDY), Spain (JSJD) and France (EURORDIS).
TEDDY as contributing entity
• TEDDY is among the few organisations whose members havebeen considered in the Consortium Agreement asContributing Entities and have rights to access to theBackground and the Results of the project at the conditionsset out in the Consortium Agreement.
• This is a very important achievement as it meansthat TEDDY members are considered in the projectas EJPRD Linked Third parties, in the same way as themembers of the involved ERICs and ERNs.
1
2 4
3FUNDING ACCELERATING
TRANSLATION OF RESEARCH &
THERAPY
DEVELOPMENT
COORDINATED
ACCESS TO
DATA &
SERVICES
CAPACITY
BUIDLING &
EMPOWERMENT
COORDINATION& TRANSVERSAL ACTIVITIES
INTEGRATIVE RESEARCH STRATEGY
SUSTAINABILITY
ETHICAL & REGULATORY
COMMUNICATION
Coordinated by
COORDINATION &TRANSVERSAL ACTIVITIES
INTEGRATIVE RESEARCH & INNOVATION STRATEGY
ETHICS, LEGAL, REGULATORY & IPR
COMMUNICATION & DISSEMINATION
SUSTAINABILITY
PROGRAMME MANAGEMENT & COORDINATION
TEDDY will be involved in the project in the communication activities. It will provide its
experience in the field of paediatrics, ensuring the right awareness on the importance of the
paediatric aspects in the field of the rare disease research.
Coordinated by
CONCLUSIONS
TEDDY will be central to further engage and empower rare disease patients intoresearch and provide them with the tools required to apprehend and activelycontribute to innovation and therapy development.
There will also be relevant links and synergies with the ongoing H2020 project EPTRIEuropean Paediatric Translational Research Infrastructure, coordinated by CVBF, theH2020 project PEDCRIN Paediatric Clinical Research Infrastructure Networkcoordinated by ECRIN and with the IMI2 C4C project.
top related